Hunter Syndrome Treatment Market – Global Industry Analysis, Trends, and Forecast
The global hunter syndrome treatment market size is projected to expand at a substantial CAGR during the forecast period, 2021–2028. The growth of the market is attributed to the introduction of novel therapies, robust product pipeline, increasing government initiatives, and rising awareness regarding hunter syndrome.
Hunter syndrome, often called mucopolysaccharidosis type II (MPS II), is a rare genetic illness caused by a lack of the enzyme iduronate-2-sulfatase. There is no permanent remedy and solution to cure the illness at present. Currently available treatments such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), are aimed at providing symptomatic relief and managing problems related to disease progression.
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Hunter syndrome affects about one in 160,000 males worldwide, according to data published by the National Institute of Health (NIH) in 2018. As the report continues, women have a lower risk of having this condition due to two X chromosomes factor, one of which can offer a working gene if the other is found deficient. In men, however, there is no other X chromosome to compensate for the one that is faulty. Two significant reasons expected to drive market expansion in the near future are expected approval of novel medicines in late-phase clinical studies and increased R&D activity by key players for the development of such novel therapeutics. Denali Therapeutics Inc., for example, received approval of their therapeutic products, which are categorized as orphan drug designation and rare pediatric disease designation from the US Food and Drug Administration (FDA) in June 2019 for its pipeline drug candidate DNL310.
Various organizations’ initiatives aimed at raising awareness about disease diagnosis and treatment are projected to drive market expansion over the predicted period. For example, in May 2018, Shire Plc. launched its third #FlyforMPS digital campaign in partnership with the National MPS Society and the International MPS Network, with the goal of raising awareness about mucopolysaccharide diseases. Among the seven types of MPS, hunter syndrome or MPS II represents the most severe disorder that can affect tissues and organs of a child over their lifetime.
Market Trends, Drivers, Restraints, and Opportunities
- Increasing government initiatives for several awareness campaigns of the disorder and encouragement for early diagnostic of the disease presents a key driving factor of the market.
- Introduction of novel therapies, wide availability of therapeutic options, and robust pipelines are anticipated to boost the market growth during the forecast period.
- Increased R&D spending and technological breakthroughs leading to the adoption of new technologies for the treatment of rare diseases are expected to spur the market growth during the forecast period.
- Requirement for prolonged treatment and high cost of the treatment are key restraining factors that can hamper the market growth.
Market Segment Insights
Enzyme replacement therapy segment is projected to hold a major market share
Based on treatments, the hunter syndrome treatment market is divided into enzyme replacement therapy, hematopoietic stem cell transplant, and others. The enzyme replacement therapy segment is expected to hold a key share of the market during the forecast period owing to wide adoption of the technique as a first-line treatment method and increasing approval of the treatment method from regulatory bodies across the globe. For instance, Shire Plc’s ELAPRASE and GC Pharma’s products are widely approved to treat the disorder. The rise in demand for these medications is due to existing need for targeted patients worldwide. The high cost of these drugs, on the other hand, remains a major impediment to the market growth. For example, the drug Idursulfase (Elaprase) is normally cost around USD 3,100 per 6mg/3ml vial.
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On the basis of regions, the market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. The market of Asia Pacific is anticipated to expand at an impressive CAGR during the forecast period owing to the high unmet needs of the treatment in the region. Major players are focusing on getting their innovative therapies approved and conquering these markets to earn a significant share. In China, for example, CANbridge Pharmaceuticals Inc. filed a New Medicine Application (NDA) with the National Medical Products Administration (NMPA) in July 2019 for Hunterase, a therapy drug. Hunterase (idursulfase beta) is a GC Pharma-developed proprietary medicine for the treatment of the disease.
Meanwhile, North America dominated the market in 2018 as a result of favorable legislation for orphan drug development, higher public awareness of rare diseases, increased financing for research, and improved healthcare facilities. Furthermore, the regional market is projected to hold a major share of the market in the coming years owing to factors such as advantageous reimbursement procedures for pricey pharmaceuticals including ELAPRASE especially in the US.
Key players competing in the global hunter syndrome treatment market are Shire Plc. (Takeda Pharmaceutical Company); GC Pharma; JCR Pharmaceuticals Co Ltd.; RegenxBio Inc.; Sangamo Therapeutics, Inc.; ArmaGen Inc; Inventiva S.A.; Denali Therapeutics Inc.; Bioasis Technologies Inc.; and Esteve.
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